IN2014DN06789A - - Google Patents

Info

Publication number
IN2014DN06789A
IN2014DN06789A IN6789DEN2014A IN2014DN06789A IN 2014DN06789 A IN2014DN06789 A IN 2014DN06789A IN 6789DEN2014 A IN6789DEN2014 A IN 6789DEN2014A IN 2014DN06789 A IN2014DN06789 A IN 2014DN06789A
Authority
IN
India
Prior art keywords
paracrine
nucleic acid
encoding nucleic
gene
individual
Prior art date
Application number
Inventor
H Kirk Hammond
Mei Hua Gao
Original Assignee
Univ California
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ California filed Critical Univ California
Publication of IN2014DN06789A publication Critical patent/IN2014DN06789A/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/861—Adenoviral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/436—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/65—Tetracyclines
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/2221—Relaxins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/2228—Corticotropin releasing factor [CRF] (Urotensin)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/2242—Atrial natriuretic factor complex: Atriopeptins, atrial natriuretic protein [ANP]; Cardionatrin, Cardiodilatin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/25—Growth hormone-releasing factor [GH-RF] (Somatoliberin)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/30—Insulin-like growth factors (Somatomedins), e.g. IGF-1, IGF-2
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/52—Isomerases (5)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6901—Conjugates being cells, cell fragments, viruses, ghosts, red blood cells or viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/06—Ointments; Bases therefor; Other semi-solid forms, e.g. creams, sticks, gels
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04—Antibacterial agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/12—Antihypertensives
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/075—Adenoviridae
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/57509—Corticotropin releasing factor [CRF] (Urotensin)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/65—Insulin-like growth factors (Somatomedins), e.g. IGF-1, IGF-2
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2121/00—Preparations for use in therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • C12N2830/003—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor tet inducible

Abstract

In alternative embodiments the invention provides methods for treating ameliorating or protecting (preventing) an individual or a patient against a disease an infection or a condition responsive to an increased paracrine polypeptide level in vivo comprising: providing a paracrine polypeptide encoding nucleic acid or gene operatively linked to a transcriptional regulatory sequence; or an expression vehicle a vector a recombinant virus or equivalent having contained therein a paracrine encoding nucleic acid or gene and the expression vehicle vector recombinant virus or equivalent can express the paracrine encoding nucleic acid or gene in a cell or in vivo; and administering or delivering the paracrine polypeptide encoding nucleic acid or gene operatively linked to a transcriptional regulatory sequence or the expression vehicle vector recombinant virus or equivalent to an individual or a patient in need thereof thereby treating ameliorating or protecting (preventing) the individual or patient against the disease infection or condition responsive to an increased paracrine polypeptide level.
IN6789DEN2014 2012-02-14 2013-02-13 IN2014DN06789A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261598772P 2012-02-14 2012-02-14
PCT/US2013/025997 WO2013123094A2 (en) 2012-02-14 2013-02-13 Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions

Publications (1)

Publication Number Publication Date
IN2014DN06789A true IN2014DN06789A (en) 2015-05-22

Family

ID=48984885

Family Applications (1)

Application Number Title Priority Date Filing Date
IN6789DEN2014 IN2014DN06789A (en) 2012-02-14 2013-02-13

Country Status (18)

Country Link
US (3) US20150118287A1 (en)
EP (2) EP3311830A1 (en)
JP (3) JP6562632B2 (en)
KR (1) KR102107482B1 (en)
CN (2) CN108310396B (en)
AU (2) AU2013221615B2 (en)
BR (1) BR112014020127A2 (en)
CA (1) CA2864100A1 (en)
DK (1) DK2814513T3 (en)
ES (1) ES2660492T3 (en)
HK (2) HK1203845A1 (en)
HU (1) HUE036640T2 (en)
IL (3) IL234007A0 (en)
IN (1) IN2014DN06789A (en)
PL (1) PL2814513T3 (en)
PT (1) PT2814513T (en)
RU (1) RU2642605C2 (en)
WO (1) WO2013123094A2 (en)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
KR102107482B1 (en) * 2012-02-14 2020-05-08 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions
US20170182129A1 (en) * 2014-04-03 2017-06-29 The Regents Of The University Of California Systemic delivery of virus vectors encoding urocortin-2 and related genes to treat diabetes-related cardiac dysfunctions and congestive heart failure
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US10480011B2 (en) * 2014-10-21 2019-11-19 University Of Massachusetts Recombinant AAV variants and uses thereof
CA2966620A1 (en) 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
EP3218484A4 (en) 2014-11-14 2018-05-30 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US10570395B2 (en) 2014-11-14 2020-02-25 Voyager Therapeutics, Inc. Modulatory polynucleotides
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
CN107531752B (en) * 2015-05-12 2022-05-10 加利福尼亚大学董事会 Peptide therapy for inflammation and fibrosis
CN108025047B (en) * 2015-05-28 2021-11-19 康奈尔大学 Adeno-associated virus mediated delivery of C1EI as a therapy for angioedema
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
KR102427379B1 (en) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. Compositions and methods for treating Huntington's disease
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
CA3035522A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
EP3526333A4 (en) 2016-10-13 2020-07-29 University of Massachusetts Aav capsid designs
AU2018210990A1 (en) * 2017-01-23 2019-08-29 Rutgers, The State University Of New Jersey Compositions for reducing sarcolipin expression and preventing and treating muscular dystrophy and cardiomyopathy and methods of use
AU2018228308B2 (en) 2017-02-28 2022-01-27 Genemedicine Co., Ltd. Anticancer composition comprising tumor-specific oncolytic adenovirus and immune checkpoint inhibitor
CN111108198A (en) 2017-05-05 2020-05-05 沃雅戈治疗公司 Compositions and methods for treating huntington's disease
JP2020518258A (en) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． Amyotrophic lateral sclerosis (ALS) treatment composition and method
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
US11760987B2 (en) 2017-09-07 2023-09-19 The Regents Of The University Of California Compositions and methods for the treatment or prevention of heart failure
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
EP3697905A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
EP3801465A4 (en) * 2018-06-11 2022-03-23 The Regents of the University of California Demethylation to treat eye disease
CN112826823B (en) * 2021-01-11 2022-03-08 中国人民解放军军事科学院军事医学研究院 Application of doxycycline in preparation of medicine for treating and/or preventing and/or relieving and/or improving myocardial muscle

Family Cites Families (61)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US5217879A (en) 1989-01-12 1993-06-08 Washington University Infectious Sindbis virus vectors
US5733542A (en) 1990-11-16 1998-03-31 Haynesworth; Stephen E. Enhancing bone marrow engraftment using MSCS
US5270192A (en) 1991-02-07 1993-12-14 Monsanto Company Biological artificial liver
US5846743A (en) 1995-02-22 1998-12-08 Brigham And Women's Hospital, Inc. Polyphoshoinositide binding peptides for intracellular drug delivery
US7361332B2 (en) 1995-03-17 2008-04-22 The Regents Of The University Of California Treating tumors using implants comprising combinations of allogeneic cells
US6214797B1 (en) * 1995-06-13 2001-04-10 The Salk Institute For Biological Studies Urocortin peptides, nucleic acid encoding same methods for using same
US5721118A (en) 1995-10-31 1998-02-24 The Regents Of The University Of California, San Diego Mammalian artificial chromosomes and methods of using same
EP0876165B1 (en) 1995-12-18 2006-06-21 Angiotech BioMaterials Corp. Crosslinked polymer compositions and methods for their use
US6025155A (en) 1996-04-10 2000-02-15 Chromos Molecular Systems, Inc. Artificial chromosomes, uses thereof and methods for preparing artificial chromosomes
US6933331B2 (en) 1998-05-22 2005-08-23 Nanoproducts Corporation Nanotechnology for drug delivery, contrast agents and biomedical implants
US5874268A (en) 1996-09-23 1999-02-23 Duke University Method of introducing exogenous compounds into cells by electroporation and apparatus for same
EP1488761A1 (en) * 1996-11-01 2004-12-22 Ark Therapeutics Limited Device for delivering a therapeutic agent to a blood vessel
US6630137B1 (en) 1997-04-28 2003-10-07 Eli Lilly And Company Activated protein C formulations
EA005157B1 (en) * 1997-05-06 2004-12-30 Те Риджентс Оф Те Юниверсити Оф Калифорния Techniques and compositions for treating heart failure and ventricular remodeling by in vivo delivery of angiogenic transgenes
US20050095227A1 (en) * 1997-07-22 2005-05-05 The General Hospital Corporation Treating heart failure
SE9704076D0 (en) 1997-11-06 1997-11-06 Holdingbolaget Vid Goeteborgs Method for permeabilization of cell structures and use thereof
US6886568B2 (en) 1998-04-08 2005-05-03 The Johns Hopkins University Method for fabricating cell-containing implants
AU762220B2 (en) * 1998-05-28 2003-06-19 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The AAV5 vector and uses thereof
US6589503B1 (en) 1998-06-20 2003-07-08 Washington University Membrane-permeant peptide complexes for medical imaging, diagnostics, and pharmaceutical therapy
US6958212B1 (en) 1999-02-01 2005-10-25 Eidgenossische Technische Hochschule Zurich Conjugate addition reactions for the controlled delivery of pharmaceutically active compounds
NZ546670A (en) * 1999-11-05 2009-02-28 Univ California Techniques and compositions for treating cardiovascular disease by in vivo gene delivery
CA2395839A1 (en) * 1999-12-27 2001-07-05 The Regents Of The University Of California Gene therapy for congestive heart failure
US6759386B2 (en) 2000-04-06 2004-07-06 Wayne P. Franco Methods of use of fibroblast growth factor, vascular endothelial growth factor and related proteins in the treatment of acute and chronic heart disease
US7442390B2 (en) 2000-06-05 2008-10-28 University Of South Florida Method for enhancing engraftment of cells using mesenchymal progenitor cells
DE60135850D1 (en) * 2000-08-04 2008-10-30 Res Dev Foundation UROCORTINE PROTEINS AND ITS USES
NZ523803A (en) * 2000-08-04 2004-08-27 Res Dev Foundation Human urocortin-related polypeptide (URP) and uses thereof to rreat a pathophysiological state, eg stress, anxiety and appetite dysfunction
EP1559777A1 (en) 2001-04-24 2005-08-03 Hokkaido Technology Licensing Office Co., Ltd. Process for maturating small heptocyte-rich colonies into liver tissue
EP2359869B1 (en) * 2001-12-17 2018-12-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor
US7192924B2 (en) * 2002-01-16 2007-03-20 The Procter & Gamble Company Corticotropin releasing factor 2 receptor agonists
US7379765B2 (en) 2003-07-25 2008-05-27 Dexcom, Inc. Oxygen enhancing membrane systems for implantable devices
US7166133B2 (en) 2002-06-13 2007-01-23 Kensey Nash Corporation Devices and methods for treating defects in the tissue of a living being
US20040151766A1 (en) 2003-01-30 2004-08-05 Monahan Sean D. Protein and peptide delivery to mammalian cells in vitro
US7241455B2 (en) 2003-04-08 2007-07-10 Boston Scientific Scimed, Inc. Implantable or insertable medical devices containing radiation-crosslinked polymer for controlled delivery of a therapeutic agent
US7794706B2 (en) 2003-10-14 2010-09-14 Medivas, Llc Bioactive wound dressings and implantable devices and methods of use
US20050136121A1 (en) 2003-12-22 2005-06-23 Shear/Kershman Laboratories, Inc. Oral peptide delivery system with improved bioavailability
JP3903061B2 (en) 2003-12-24 2007-04-11 株式会社Ｌｔｔバイオファーマ Nanoparticles containing drug, method for producing the same, and preparation for parenteral administration comprising the nanoparticles
US20050244463A1 (en) 2004-04-30 2005-11-03 Allergan, Inc. Sustained release intraocular implants and methods for treating ocular vasculopathies
US8658203B2 (en) 2004-05-03 2014-02-25 Merrimack Pharmaceuticals, Inc. Liposomes useful for drug delivery to the brain
WO2006014035A1 (en) 2004-08-06 2006-02-09 Biospectrum, Inc. Multiple layered liposome and preparation method thereof
EP1788970A2 (en) 2004-08-20 2007-05-30 Artes Medical, Inc. Methods of administering microparticles combined with autologous body components
WO2006026325A2 (en) 2004-08-26 2006-03-09 Pathak Chandrashekhar P Implantable tissue compositions and method
US7351423B2 (en) 2004-09-01 2008-04-01 Depuy Spine, Inc. Musculo-skeletal implant having a bioactive gradient
US7501486B2 (en) 2004-09-07 2009-03-10 Burnham Institute For Medical Research Peptides that selectively home to heart vasculature and related conjugates and methods
DE602005025676D1 (en) 2004-10-18 2011-02-10 Nitto Denko Corp INTRA-CELLULAR PEPTIDE DELIVERY
EP1885746B1 (en) * 2005-02-08 2012-01-11 Research Development Foundation Compositions related to soluble g-protein coupled receptors (sgpcrs)
US7759120B2 (en) 2005-03-02 2010-07-20 Kps Bay Medical, Inc. Seeding implantable medical devices with cells
US20070059288A1 (en) * 2005-03-31 2007-03-15 Dinsmore Jonathan H Treatment for heart disease
EP1937213B1 (en) 2005-07-27 2017-10-25 Protiva Biotherapeutics Inc. Systems and methods for manufacturing liposomes
US20070265221A1 (en) 2006-05-09 2007-11-15 Weiss Robert G Methods to improve creatine kinase metabolism and contractile function in cardiac muscle for the treatment of heart failure
WO2008047241A2 (en) * 2006-10-16 2008-04-24 Conjuchem Biotechnologies Inc. Modified corticotropin releasing factor peptides and uses thereof
CA2580907A1 (en) 2006-11-21 2008-05-21 The Bionic Ear Institute Choroid plexus cell implantation to prevent and/or treat hearing loss
EP2209889B1 (en) * 2007-11-16 2016-07-20 San Diego State University Research Foundation Compositions for manipulating pim-1 activity in cardiac cells
AU2008346801A1 (en) * 2007-12-31 2009-07-16 Nanocor Therapeutics, Inc. RNA interference for the treatment of heart failure
BRPI0912559A2 (en) * 2008-05-16 2015-10-13 Corthera Inc method of treating chronic heart failure
CN106177914A (en) * 2008-05-16 2016-12-07 科尔泰拉公司 With the relaxin treatment dyspnea relevant to acute heart failure
CA2742710A1 (en) * 2008-11-04 2010-05-14 Janssen Pharmaceutica Nv Crhr2 peptide agonists and uses thereof
WO2010138263A2 (en) * 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
JP2013510167A (en) * 2009-11-04 2013-03-21 ヤンセン ファーマシューティカ エヌ．ベー． Treatment of heart failure with stress-copin-like peptides
KR102107482B1 (en) * 2012-02-14 2020-05-08 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions
KR20180102172A (en) * 2016-01-19 2018-09-14 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 How to treat disability and other self-predisposing disorders

Also Published As

Publication number Publication date
EP2814513A4 (en) 2016-01-13
US20150118287A1 (en) 2015-04-30
AU2018200719B2 (en) 2019-10-24
HUE036640T2 (en) 2018-07-30
RU2014137169A (en) 2016-04-10
JP2019135263A (en) 2019-08-15
US20190367945A1 (en) 2019-12-05
DK2814513T3 (en) 2018-02-26
EP2814513B1 (en) 2017-12-20
JP2015508760A (en) 2015-03-23
CA2864100A1 (en) 2013-08-22
CN104220098B (en) 2018-04-10
KR20140124403A (en) 2014-10-24
CN108310396A (en) 2018-07-24
KR102107482B1 (en) 2020-05-08
IL262600A (en) 2018-12-31
IL262600B (en) 2019-11-28
IL234007A0 (en) 2014-09-30
HK1254639A1 (en) 2019-07-26
JP6562632B2 (en) 2019-08-21
IL261172A (en) 2018-10-31
ES2660492T3 (en) 2018-03-22
EP2814513A2 (en) 2014-12-24
US10202618B2 (en) 2019-02-12
PL2814513T3 (en) 2018-06-29
US10918738B2 (en) 2021-02-16
HK1203845A1 (en) 2015-11-06
AU2018200719A1 (en) 2018-02-22
CN108310396B (en) 2022-07-15
BR112014020127A2 (en) 2020-06-30
JP2017203048A (en) 2017-11-16
AU2013221615B2 (en) 2018-02-22
WO2013123094A2 (en) 2013-08-22
PT2814513T (en) 2018-03-02
US20180010148A1 (en) 2018-01-11
EP3311830A1 (en) 2018-04-25
AU2013221615A1 (en) 2014-08-28
RU2642605C2 (en) 2018-01-25
CN104220098A (en) 2014-12-17

Similar Documents

Publication Publication Date Title
IN2014DN06789A (en)
NZ704275A (en) Aav-mediated gene therapy for rpgr x-linked retinal degeneration
MX2016002152A (en) Method for increasing expression of rna-encoded proteins.
NZ598348A (en) Combined use of gdf traps and erythropoietin receptor activators to increase red blood cell levels
BR112016028023A2 (en) COMPOSITIONS AND METHODS OF ADMINISTRATION OF TREATMENTS FOR LATENT VIRAL INFECTIONS
BR112016016290A2 (en) PHARMACEUTICAL COMPOSITIONS OF FAST-ACTING INSULIN, INFUSION SET, INFUSION SET SYSTEM AND USES OF MONOMER OR DIMERIC INSULIN ANALOGS
BR112014019901A8 (en) RECOMBINANT FACTOR VIII PROTEINS
MX357803B (en) Artificial nucleic acid molecules.
ZA201506093B (en) Methods and pharmaceutical composition for the treatment and the prevention of cardiomyopathy due to energy failure
MX344727B (en) IMPROVED HIGH CONCENTRATION ANTI-TNFa ANTIBODY LIQUID FORMULATIONS.
NZ587105A (en) Tissue protective peptides and peptide analogs for preventing and treating diseases and disorders associated with tissue damage
MA40057A (en) Therapeutic tratment of skin disease with recombinant commensal skin microorganisms
IN2014DN09554A (en)
IN2014DN06920A (en)
PH12014501388A1 (en) A recombinant koi herpesvirus (khv) and vaccine for the prevention of a disease caused by khv
MX366134B (en) Angiotensin in treating brain conditions.
MX2016012558A (en) Systemic delivery of virus vectors encoding urocortin-2 and related genes to treat diabetes-related cardiac dysfunction and congestive heart failure.
MX2014002062A (en) Peptide nanoparticles and uses thereof.
EA201790178A1 (en) PEPTIDES BEARING MANNOSE-6-PHOSPHATE, FUSED WITH LIZOSOMAL ENZYME
EP2552462A4 (en) Ccn3 peptides and analogs thereof for therapeutic use
MX2009003636A (en) Novel peptides for treating and preventing immune-related disorders, including treating and preventing infection by modulating innate immunity.
MY168778A (en) Oligonucleotide chelate complex-polypeptide compositions and methods
IN2014DN09963A (en)
NZ603442A (en) Treatment of proliferative diseases
MX2016007950A (en) T cell receptor binding to alwgpdpaaa, derived from human pre-pro insulin (ppi) protein.